Articles tagged with Gene Therapy
Researchers from the University of Pennsylvania have shown that early exposure to the human IDUA protein increased immune tolerance, allowing for widespread expression of the enzyme and resolution of brain lesions. The study informs the planning of first-in-human trials for MPS I treatment.
The TAILOR-PCI Study aims to determine whether prescribing antiplatelet therapy based on a patient's CYP2C19 genotype can prevent heart attack, stroke, and cardiovascular death. The study, which began in 2013, plans to enroll 5,270 patients and investigate the effects of genomics on treatment outcomes.
A new gene therapy approach has shown promising results in treating hereditary tyrosinemia type 1 (HT1), a metabolic disorder characterized by progressive liver disease. The treatment involves transplanting corrected liver cells into the diseased liver, resulting in enzyme production and preventing liver failure.
Researchers have developed a gene delivery system that can be applied as an eye drop, rapidly moving from the surface to the retina and remaining for over eight hours. This method shows promise for treating several eye diseases, including diabetic retinopathy and age-related macular degeneration.
A new study found no serious adverse events related to gene therapy in patients with advanced Parkinson's disease five years after treatment. The long-term safety profile of the therapy is promising, lending support to its potential as a treatment for this debilitating disease.
Researchers have identified three new risk genes for amyotrophic lateral sclerosis (ALS) using genetic data from Project MinE. One of these genes, C21orf2, increases the risk of developing ALS by 65%. The study's findings could aid the development of personalized treatments using gene therapy.
A recent study by Sanford Health identified gene mutations that could impact treatment for adult cancer patients. The study found nearly 90% of patients had genes that matched treatment options, with 40% able to receive personalized therapy.
Researchers describe expanding scope of gene therapy targeting CNS diseases, including Alzheimer's and ALS. The article highlights rapid progress in viral vector development and delivery strategies for treating these conditions.
Researchers have identified three new genes that increase the risk of motor neuron disease (MND), a debilitating condition with no effective treatments. The discovery provides new opportunities for targeted research and potentially improved outcomes for Australian patients, who are expected to benefit from increased genetic discoveries.
Australian researchers have developed a novel gene therapy that targets muscle cells to prevent cachexia, a condition of weight loss and frailty associated with severe muscle wasting. The approach has shown promise in pre-clinical mouse models, suggesting potential benefits for treating frailty in other chronic illnesses.
A new study discovered 172 novel cancer gene-drug combinations that selectively kill cancer cells without harming healthy cells. This breakthrough expands the number of actionable mutations and provides a promising approach to personalized cancer therapy.
A new study has developed a methodology to analyze bacterial virulence factors and identify therapeutic targets for developing effective antimicrobial agents. The TREP approach was applied to Haemophilus influenzae, a respiratory pathogen that causes COPD symptoms, revealing potential blocking elements for invasion.
Researchers have successfully programmed a virus to transport genetic material to affected tissue and nerve cells, opening up new options for treating various diseases. The approach uses attenuated viruses to selectively target diseased cells, reducing damage to healthy ones.
Researchers have developed a technique to program stem cells to grow new cartilage on a 3-D template shaped like the ball of a hip joint. The cartilage can release anti-inflammatory molecules to fend off arthritis. The discovery may provide an alternative to hip-replacement surgery, particularly in younger patients.
A study at the University of Texas MD Anderson Cancer Center found that genetic mutations in DNA mismatch repair deficiency (dMMR) are present in 15% of colorectal cancer cases. The study identified MSH2 and MSH6 as the most commonly affected genes, providing a new approach to diagnosis and treatment using precision medicine.
Researchers have found that chromatin remodeling proteins, essential for DNA replication and gene expression, are frequently mutated in human cancers. These mutations indicate tumor suppressive functions, making chromatin remodelers promising new targets for targeted cancer therapy.
A new study from TSRI researchers reveals the role of a gene called Mkx in maintaining and strengthening tendons in animal models. The findings may bring doctors closer to using gene therapies to grow and repair tendons.
Scientists at Northwestern University have identified a novel target for depression treatment by manipulating HCN channels in the hippocampus. Reducing these channel's function has been shown to improve mood in mice, offering new hope for millions of patients who don't respond to existing treatments.
A clinical isolate of bacterial pathogen carrying the colistin resistance gene mcr-1 has been detected in humans in the US, sparking concerns about antibiotic resistance. The isolate was found to be susceptible to several commonly used antimicrobial agents, but its plasmid-mediated nature is still unknown.
Researchers have developed a lung-targeted gene therapy strategy that may improve treatments for inherited diseases like emphysema. The new approach, which delivers a normal copy of the gene to lung cells, shows promise in reducing disease severity and increasing protein production.
Researchers developed an anti-apoE4 specific monoclonal antibody that reverses key brain-related pathological effects of apoE4 and associated cognitive deficits in mice. The treatment also accumulates in the brain and binds to apoE4, suggesting a novel approach for AD therapy.
Researchers identified a network of genes shared by CLL patients and associated with patient survival, which could be targets for treatment. Epigenetic analysis is expected to become widely used for selecting personalized treatments in cancer and other diseases.
Brendan Lee receives 2016 Curt Stern Award for his groundbreaking work on human inborn errors of metabolism and structural birth defects of the skeleton. The award recognizes his significant scientific contributions over the past decade, including identifying genetic causes of chrondrodysplasia and Marfan syndrome.
Researchers have identified a genetic mutation in the TEK gene that causes primary congenital glaucoma, a devastating eye disease affecting children worldwide. The finding validates previous research in mice and suggests a new therapeutic pathway to repair damaged vessels in the eye.
A new hybridization-induced aggregation technology detects KRAS mutations in lung and colorectal cancers, enabling targeted therapies. The technique is simple, rapid, and inexpensive, with 100% agreement with sequencing results.
Researchers develop new method to generate large pool of gene-corrected cells, which can repopulate in vivo and treat various disorders
A University of Colorado Cancer Center study used whole-exome sequencing to characterize genetic alterations in 25 commonly used bladder cancer cell lines. The researchers found 76 altered genes, many involving oncogenes like TERT and TP53, which can predict patient response to cisplatin treatment
Researchers have identified a protein called TST that helps detoxify waste products in fat cells, leading to improved insulin sensitivity and reduced weight gain in obese mice. The study's findings suggest a potential new approach to treating type 2 diabetes associated with obesity.
A new study discovered a genetic variant that reduces the risk of coronary heart disease in people taking antidiabetic medications, contradicting previous assumptions about these drugs' safety. The variant was linked to the glucagon-like peptide-1 receptor and may provide valuable insights for predicting drug side effects earlier in th...
Researchers have discovered a combination of cells and genes that can repair damaged heart tissues in animal models of myocardial infarction. The study found that different biological treatments target various aspects of cardiac function and scarring, with some showing significant improvements in contractile function and angiogenesis.
A team of scientists has identified the Sostdc1 gene as a key regulator of periosteum stem cells during fracture repair, which could lead to new therapeutic treatments for difficult-to-heal injuries. The study found that mutant mice lacking the gene had thicker, denser cortical bone that healed at an accelerated rate.
Recent evidence suggests that AAV vectors used for gene therapy can trigger liver cancer in rare patients. A new article challenges re-interpreted data, reaffirming the link between insertional mutagenesis and hepatocellular carcinoma.
Researchers have developed a novel therapeutic strategy for treating cystic fibrosis, restoring lung cell function to 50% of healthy levels. By using a yeast genetic model, they identified key targets that can rescue the misfolding of the deltaF508-CFTR protein, which affects nearly 90% of patients with cystic fibrosis.
A new gene therapy has been developed to repair lungs damaged by chronic allergic asthma, reducing inflammation and improving airway structure and function. The study used a novel biodegradable platform to deliver the thymulin gene therapy, showing effective results in mice with experimental asthma.
Researchers at SISSA have developed a gene therapy approach targeting glioblastomas by introducing an active version of the Emx2 gene, which inhibits astrocyte growth and leads to tumour cell suicide. The treatment has demonstrated efficacy in both vitro and in vivo tests, with potential for preventing aggressive recurrence development.
Researchers evaluated an IHC screening tool for ROS1 gene rearrangements in a cohort of 170 patients. The results showed high sensitivity and specificity rates, making it a feasible option for first-line screening in a lung cancer setting.
Researchers found that genetically modifying oncolytic viruses to express a junction opening (JO) protein significantly enhances their ability to penetrate and kill malignant epithelial cells. This breakthrough could improve cancer therapy for bulkier solid tumors.
Researchers develop an experimental therapy that shuts down the Olig2 gene, halting tumor growth and blocking tumor formation. The approach uses a gene therapy to eliminate Olig2-positive cells, sensitizing them to targeted molecular treatment.
Researchers discuss zebrafish as a model system for studying normal hematopoiesis and blood disorders. The review highlights the knowledge gained from studies of zebrafish, including large-scale forward genetic screens and modeling of human genes to create transgenic zebrafish.
Researchers at the University of Missouri have developed a gene replacement therapy to treat and control Spinal Muscular Atrophy with Respiratory Distress Type 1 (SMARD1). The therapy, administered in a single dose, has shown significant improvements in muscle strength, protein expression, and life span in SMARD1 mice. This study provi...
Researchers at UT Southwestern Medical Center discovered that Fanconi anemia genes play a crucial role in selective autophagy, particularly in removing damaged mitochondria. This finding has implications for treating Fanconi anemia and certain cancers, including familial breast and ovarian cancer.
Scientists used gene therapy to halt pulmonary hypertension progression in a large animal model. The approach delivered SERCA2a gene via aerosol inhalation, slowing vascular changes and improving heart function. Further studies are needed to assess long-term efficacy and safety before human clinical trials.
A pioneering gene therapy has restored vision to patients with a rare form of genetic blindness for up to four years, providing sustained improvement and raising hopes for a cure. The treatment involves injecting a virus into the eye to deliver billions of healthy genes, replacing a key missing gene.
Researchers developed a method demonstrating that independent DNA variants linked to a disease share similar biological properties. This discovery provides a more comprehensive picture of disease biology and offers the opportunity to broaden our understanding of disease and identify new therapeutic targets.
Researchers at Lund University have found a promising new therapeutic target to treat bacterial infections, regulating the immune response and avoiding destructive inflammation. The discovery of IRF-7 as an immunotherapeutic target holds promise for non-antibiotic therapy.
A new study reveals that a genetic risk factor for five major psychiatric diseases may be linked to the death of newborn brain cells, which can disrupt learning, mood, and anxiety. The researchers found that a compound currently being developed could potentially prevent this cell death and have therapeutic value for these diseases.
Autologous cardiac cells, particularly atrial appendage-derived cells, show promise for gene and RNA therapies in heart failure. The review discusses financial and authority-based restrictions influencing research in this field, highlighting the potential of autologous tissues as cell therapy sources.
A biologically younger woman demonstrates the world's first successful gene therapy against human aging. Elizabeth Parrish's white blood cells showed a 20-year increase in telomere length, implying a reversal of age-related diseases.
A novel gene therapy developed at St. Jude Children's Research Hospital has shown promising results in rebuilding the immune system of young adults with X-linked severe combined immunodeficiency disease (SCID-X1). The treatment, which combines lentivirus gene therapy with busulfan conditioning, resulted in improved health and reduced c...
Researchers have discovered a novel host defense mechanism that protects cells from viral infection by sequestering virions near the nucleus. This discovery has implications for new drug discovery and therapeutic gene delivery, and may lead to improved strategies for rAAV-based gene therapy.
Dr. Crystal Mackall receives $500,000 Clinical Translation Grant to study osteosarcoma and develop immunotherapy treatment options. The grant aims to improve outcomes for patients with this rare form of cancer.
A new study in patients with methylmalonic acidemia found lower than expected levels of antibodies against adeno-associated viral vectors, suggesting encouraging implications for gene delivery as a treatment. The absence of AAV antibodies in most patients who lack the deficient enzyme has promising results for gene therapy.
Pediatric researchers identified a new syndrome causing intellectual disability, linked to a mutation in the TBCK gene. A potential targeted treatment using an amino acid supplement has been proposed based on cell studies.
Scientists at EMBL have increased the efficiency of gene therapy tool Sleeping Beauty, which is being trialed for therapies targeting B-cell leukaemias and lymphomas. The enhancement results in a 30% increase in efficiency and aims to improve patient outcomes.
A study published in JAMA Cardiology found that intracoronary gene transfer increased left ventricular function beyond standard heart failure therapy. The treatment, which involves introducing a specific protein into heart cells, showed significant benefits in patients with nonischemic heart failure.
Researchers have found that suppressing the nuclear receptor protein ROR-γ with small-molecule compounds can reduce androgen receptor levels in castration-resistant prostate cancer, stopping tumor growth. This novel approach targets the root cause of the problem - the overexpression of the AR gene and its protein.
A global study has identified hundreds of genes associated with 5 common inflammatory diseases, paving the way for new treatments. The research found that these conditions share similar genetic backgrounds and can occur together due to shared risk factors.
A new Scripps Research Institute study has identified specific genetic variations associated with increased susceptibility to bipolar disorder and other conditions. The research focuses on a gene called PDE10A, which produces proteins that regulate intracellular levels of cAMP, influencing biological processes like learning and memory.
Researchers discovered that healthy carriers of a rare respiratory disease gene have enhanced respiratory capacity and increased height, with a 10% advantage. The study may lead to new treatment approaches for physical fitness training and lung function disorders, as well as height modification in growth disorders.
Researchers have identified two genes that, when altered, promote or undermine cardiovascular health. ANGPTL4 is associated with lower triglycerides and reduced risk of coronary disease, suggesting strategies to lower triglycerides could help prevent heart attacks. Future therapeutics targeting this pathway are in development.